ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation

NCT ID: NCT04702360

Last Updated: 2021-07-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

APPROVED_FOR_MARKETING

Study Classification

EXPANDED_ACCESS

Brief Summary

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The purpose of this program is to provide elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) to CF patients in critical need who are 6 to 11 years of age with at least one F508del mutation in response to unsolicited physician requests.

Detailed Description

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Conditions

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Cystic Fibrosis

Interventions

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ELX/TEZ/IVA

Fixed-dose combination tablet for oral administration.

Intervention Type DRUG

IVA

IVA monotablet for oral administration.

Intervention Type DRUG

Other Intervention Names

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elexacaftor/tezacaftor/ivacaftor VX-445/VX-661/VX-770 ivacaftor VX-770

Eligibility Criteria

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Inclusion Criteria

* Patients with confirmed diagnosis of CF who have an F/any genotype
* Prior treatment for at least 6 months with an approved alternative CFTR modulator (if the patient is eligible for such treatment)
* Patients with percent predicted forced expiratory volume in 1 second (ppFEV1) \<40 for a minimum of 60 consecutive days before the date of completion of the request form

Exclusion Criteria

* Patients with severe hepatic impairment
Minimum Eligible Age

6 Years

Maximum Eligible Age

11 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Other Identifiers

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VX20-445-905

Identifier Type: -

Identifier Source: org_study_id

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